A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries

Abstract

Background: Despite international initiatives on collaboration within the field of rare diseases, patient access to orphan medicinal products (OMPs) and healthcare services differ greatly between countries. This study aimed to create a comprehensive and in-depth overview of rare diseases policies and reimbursement of OMPs in a selection of 12 countries in the Western Eurasian region: Armenia,

Access to Affordable Orphan Medicines in Europe: An EHA Position Paper

“…A survey conducted in 12 Eurasian countries on rare disease policies and orphan drug reimbursement systems showed that inequality in patient access to new OMPs still exists due to variations in national healthcare budgets, health insurance, and reimbursement systems. 3 Fragmentation of reimbursement policies translates into unequal access to treatment. The number of reimbursed OMPs ranged from >100 in Germany, France, Italy, and the Netherlands to zero in Armenia.…”

Current state of developing advanced therapies for rare diseases in the European Union

“…Several EU countries have established special pathways to overcome the challenges in the HTA of ODs. For example, ultra-ODs may be eligible to undergo Highly Specialized Technology evaluations in England, which raises the ICER threshold to £100,000 per QALY gained [40]. Approved ODs were presumed to have additional benefit by law, granting with reimbursement along with MA automatically in Germany [41].…”

Price-Cutting Trends in New Drugs after Listing in South Korea: The Effect of the Reimbursement Review Pathway on Price Reduction

“…In this situation, patients can also benefit from taking advantage of innovative new drugs to treat their disease, which makes for a virtuous cycle [3]. However, the payer, who administers public insurance, has to take the effective management of budgets into account, owing to the financial burden caused by an increase in pharmaceutical expenditure [9][10][11][12][13][14]. In other words, the health technology assessment (HTA) body, which ought to efficiently distribute limited resources regarding the reimbursement and pricing of new drugs, needs to consider not only patients' access, but also the budget impact of new drugs.…”

“…In this situation, when the price is high, such problems may cause more serious issues. High-price drugs are often reimbursed because of social necessity even if the clinical outcome is uncertain; however, an unexpected increase in patient numbers may have a considerable impact on budgets [10][11][12][13][14][15][16]. Pharmaceutical companies need to raise prices to generate expected profits, while governments become more cautious about whether to reimburse or not, due to the financial burden of allocating a large amount of money to a small number of patients.…”